United States: CMS Innovation Center Announces Sickle Cell Disease Focus Under Cell And Gene Therapy Access Model

On January 30, the Centers for Medicare & Medicaid Services' (CMS') Innovation Center announced that sickle cell disease (SCD) will be the first focus of the Cell and Gene Therapy (CGT) Access Model, a model for eligible states and pharmaceutical manufacturers designed to improve Medicaid beneficiaries' access to cell and gene therapies.

Key takeaways

• The CGT Access Model is a multi-year voluntary model for states and manufacturers. The model populations include Medicaid and Children's Health Insurance Program (CHIP) beneficiaries in fee-for-service and Medicaid managed care. It will begin in 2025, with an initial focus on SCD, and could expand to other conditions.
• The CGT Access Model will test outcomes-based agreements (OBAs) for cell and gene therapies. An OBA ties payment to the manufacturer to patients' health outcomes over a certain time period. The model would seek to expand access and reduce the cost of cell and gene therapies, while also reducing the administrative and financial burden on states to administer the OBAs on their own.
• States and manufacturers should visit CMS' website to gauge interest in participating in the model. In the coming months, CMS expects to release further details on eligibility and participation. CMS will host webinars on February 6 to provide an overview of the CGT Access Model and on February 8 to provide an overview to interested states.

Initial Focus on Sickle Cell Disease

CMS states that the CGT Access Model will focus initially on expanding access to gene therapy for SCD. SCD is a genetic blood disorder, which disproportionately impacts Black Americans and causes a number of long-term health complications. Approximately 50-60% of people living with SCD are enrolled in Medicaid and hospitalizations and other health episodes related to SCD cost the health system almost $3 billion per year. Patients with SCD also experience challenges with access to quality and affordability of care. CMS states that the CGT Access Model has the potential to help improve health outcomes for SCD patients while lowering health care costs. It also states that gene therapies for the treatment of SCD can pose challenges to state budgets due to the high cost of the therapy.

Overview of the Cell and Gene Therapy Access Model

CMS first announced the CGT Access Model in February 2023 to promote increased access to drug therapies, which was developed in response to an Executive Order that President Biden issued directing the Department of Health and Human Services (HHS) to consider developing models that increase access to novel therapies and lower the high cost of drugs. Cell and gene therapies are a growing class of transformative, one-time medicines designed to treat previously intractable diseases. They often have a high upfront cost, which poses a barrier to accessing these therapies.

The CGT Access Model would create a new financing approach for patients enrolled in Medicaid to receive cell and gene therapies to which they may not have had access. Under the model, state Medicaid agencies would assign CMS to coordinate and administer multi-state, OBAs with manufacturers, which would tie pricing and align incentives for SCD treatments to whether the therapy improves health outcomes for Medicaid beneficiaries. Instead of each state creating its own OBAs with manufacturers, the CGT Access Model will allow CMS to bring states together and negotiate on a multi-state basis. Specifically, CMS provides the following details:

• Eligible Participants: The CGT Access Model allows voluntary participation from states and pharmaceutical manufacturers. Providers will not be eligible to participate in the model. All states and territories that participate in the Medicaid Drug Rebate Program (MDRP) can participate in the model if they meet requirements. CMS clarifies that if additional disease conditions are added to the model in future years, states will be permitted to apply for model participation pertaining to that condition. In addition, manufacturers who participate in the MDRP and market U.S. Food & Drug Administration (FDA)-approved or -licensed gene therapies for the treatment of severe SCD are also eligible to participate in the model.

• Model Details and Process: Under the CGT Access Model, CMS will negotiate on behalf of states the contract between states and manufacturers, which will be structured as a supplemental rebate agreement. This agreement would include pricing (that is reflective of rebates paid by the manufacturer) and outcome measures that would form the basis for individual contracts between the manufacturer and participating states. State Medicaid agencies would decide whether to sign the negotiated contract. If agreed, manufacturers will be obligated to provide states with supplemental rebates that reflect model-negotiated terms. Participating states would then be obligated to implement an agreed-upon standard access policy. States and manufacturers will have the option to include separate CHIP programs that will be subject to different considerations.

CMS will provide support through technical assistance and funding to participating states. Technical assistance would include supporting the implementation, data collection, and reconciliation of the agreements and other model activities. CMS would form a cooperative agreement with states to provide funding support, which would help states cover the costs of participation in the model, including the state obligations related to provider requirements, data collection, and activities to ensure equitable access to care. CMS will also be responsible for establishing financial and clinical outcome measures, reconciling data, and evaluating results.

• Optional State Funding: CMS anticipates addressing additional care delivery gaps and other hurdles for people receiving cell and gene therapy during the OBA negotiation process, including requiring manufacturers to include a defined scope of fertility preservation services when individuals receive gene therapy for treatment of SCD. To address these issues, CMS will offer optional funding to states that engage in activities that increase equitable access to cell and gene therapies and promote multi-disciplinary, comprehensive care for Medicaid patients with SCD receiving gene therapy. These activities may include expanding or increasing reimbursement rates for optional Medicaid benefits and services, such as behavioral health or care management services. States may apply to the Notice of Funding Opportunity (NOFO) to receive optional funding under the model.

• Application and Timeline: In March 2024, CMS will release its Manufacturer Request for Application (RFA), and manufacturers will be able to apply to the model by responding to the RFA by May 2024. States will be able to express their intent to participate by submitting a non-binding Letter of Intent (LOI) by April 2024. Negotiations between CMS and manufacturers are scheduled to take place between May and November 2024. States may apply to the model by responding to an RFA by February 2025. After states sign an agreement with CMS, states may begin participation in the model between January 2025 and January 2026. The CGT Access Model will operate for up to approximately 11 years, depending on the OBA term for each state.

Conclusion

The recent announcement on the initial SCD focus of the CGT Access Model demonstrates CMS' commitment to advance health equity, in alignment with the CMS Framework for Health Equity. Many SCD patients are from underserved communities with limited access to specialized care and experience a host of health disparities. By increasing access to therapies and offering funding to states to reduce access barriers for SCD patients, CMS states that the CGT Access Model can help address historic disparities, poor health outcomes, and low life expectancy associated with SCD. CMS has also stated that it may include additional cell and gene therapies for other diseases in the model at a future time.

The content of this article is intended to provide a general guide to the subject matter. Specialist advice should be sought about your specific circumstances.